Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Huntingtin Haplotypes Provide Prioritized Target Panels for Allele-specific Silencing in Huntington Disease Patients of European Ancestry.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Nov 2015

Kay Chris, Collins Jennifer A, Skotte Niels H, Southwell Amber L, Warby Simon C, Caron Nicholas S, Doty Crystal N, Nguyen Betty, Griguoli Annamaria, Ross Colin J, Squitieri Ferdinando, Hayden Michael R

Abstract excerpt

Huntington disease (HD) is a dominant neurodegenerative disorder caused by a CAG repeat expansion in the Huntingtin gene (HTT). Heterozygous polymorphisms in cis with the mutation allow for allele-specific suppression of the pathogenic HTT transcript as a therapeutic strategy. To prioritize target selection, precise heterozygosity estimates are needed across diverse HD patient populations. Here we present the...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Gene Targeting
Share to Genetic Therapy

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Huntingtin Protein

Share to Huntington Disease

Share to INDEL Mutation

Share to Nerve Tissue Proteins

Share to Oligonucleotides, Antisense

Share to Trinucleotide Repeat Expansion

Source

Open in PubMed Open full text

Identifiers

PMID: 26201449
DOI: 10.1038/mt.2015.128
PMCID: PMC4817952

Genes

HTT

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 3 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Huntingtin Haplotypes Provide Prioritized Target Panels for Allele-specific Silencing in Huntington Disease Patients of European Ancestry.PMID 26201449Read ↗

A Comprehensive Haplotype-Targeting Strategy for Allele-Specific HTT Suppression in Huntington Disease.Semantic proximity Read ↗

Personalized gene silencing therapeutics for Huntington disease.Semantic proximity Read ↗

Distribution of the HTT Gene A1 and A2 Haplotypes Worldwide: A Systematic Review.Semantic proximity Read ↗

Sequence-Level Analysis of the Major European Huntington Disease Haplotype.Semantic proximity Read ↗

Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin.Semantic proximity Read ↗

A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference.Semantic proximity Read ↗

Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS.Semantic proximity Read ↗

Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients.Semantic proximity Read ↗

An insight into allele-selective approaches to lowering mutant huntingtin protein for Huntington's disease treatment.Semantic proximity Read ↗

Selective targeting of mutant <i>huntingtin</i> intron-1 improves rescue provided by antisense oligonucleotidesSemantic proximity Read ↗

Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat.Semantic proximity Read ↗

Transfer of genetic therapy across human populations: molecular targets for increasing patient coverage in repeat expansion diseases.Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.